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Cystic fibrosis (CF) remains the most common lethal disease associated with a single gene defect in populations of European descent. The gene that prevents CF is the cystic fibrosis transmembrane conductance regulator (CFTR), an ATP-dependent anion channel expressed mostly at the apical surface of epithelia lined with mucus secretions. CF results from a deficiency in CFTR amount and/or function. Fortunately,...
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